KROS is advancing KER-065 for Duchenne muscular dystrophy, with orphan drug status and a phase II trial planned for early 2026.

Duchenne muscular dystrophy (DMD) is a genetic condition that primarily affects people assigned male at birth. However, due to the condition’s X-linked inheritance pattern, only people assigned female ...

The US Food and Drug Administration (FDA) has approved the antisense oligonucleotide casimersen (Amondys 45, Sarepta Therapeutics) injection for the treatment of patients with Duchenne muscular ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice. If efficiently and safely scaled up ...

Barry Byrne, MD, PhD, Powell Gene Therapy Center at the University of Florida, discusses gene therapy considerations for pediatric patients and how newborn screening can influence outcomes for ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended renewal of conditional marketing authorization for ataluren (Translarna, PTC Therapeutics) for ...

Upgrading Dyne Therapeutics to 'Buy' due to promising late-stage DM1 and DMD drug candidates and significant unmet need in both indications. Recent management changes and a more measured clinical ...